What are Lysosomal Storage Disorders (LSD’s)
Lysosomes are the body’s cleaning system. In normal function, waste materials at a cellular level are processed in the lysosomes, “recycled” by enzymes and broken down into simpler components for the cells to utilize. Lysosomal storage disorders, simply put, result when certain enzymes are not present. The result is that certain proteins and other byproducts accumulate and become toxic to the body. Depending on the type of deficiency, different body systems are affected.
There are currently over 50 identified lysosomal storage disorders identified, including Fabry disease, Gaucher disease, Hurler syndrome, Niemann-Pick types A, B, and C, Pompe disease and Tay-Sachs disease. All lysosomal storage diseases are genetic and individually extremely rare.
Our thinking is that, although each lysosomal storage disorders is different, many share common hurdles to overcome. For example, crossing the blood/brain barrier has been a tremendous challenge in creating potential therapies. We strongly believe that, by focusing on the common obstacles, the answers for one lysosomal storage disease may be the key to others as well.
TODAY 48 BABIES WILL BE BORN WITH A LYSOSOMAL STORAGE DISORDER
Lysosomal Storage Fast Facts
- Individually Lysosomal Storage Disorders are extremely rare, but combined they effect roughly 1 in 7000 children.
- There are more than 50 identified LSD’S, most of which have no treatment options and are fatal by early childhood.
- Every 30 minutes a child is diagnosed with a Lysosomal Storage Disorder, and most wont live to see their 5th birthday.
- Currently there are no cures for these devastating diseases, and only 6 of them have an FDA approved treatment